FDA Accepts Priority Review of Sibeprenlimab BLA for IgAN Treatment

27 May 2025
Otsuka Pharmaceutical Co., Ltd., along with its subsidiary Otsuka Pharmaceutical Development & Commercialization, Inc., has announced a significant development regarding sibeprenlimab, a monoclonal antibody designed to aid patients with immunoglobulin A nephropathy (IgAN). The U.S. Food and Drug Administration (FDA) has accepted their Biologics License Application (BLA) for review. Sibeprenlimab works by selectively inhibiting APRIL (A PRoliferation-Inducing Ligand), a critical factor in the development of IgAN. IgAN, a chronic kidney disease, often progresses to end-stage kidney disease (ESKD).

This novel treatment offers patients a user-friendly single-dose prefilled syringe for subcutaneous injection, designed for self-administration at home every four weeks. The approach aims to provide convenience and improve quality of life for those managing this chronic condition.

The decision by the FDA to review the BLA is supported by evidence from two key clinical trials: the Phase 3 VISIONARY trial and the Phase 2 ENVISION trial. The VISIONARY trial demonstrated that sibeprenlimab effectively reduced 24-hour urinary protein-to-creatinine ratio (uPCR) over nine months compared to a placebo, meeting the primary endpoint. These significant findings have led to a priority review status, with a Prescription Drug User Fee Act (PDUFA) target action date set for November 28, 2025.

Dr. John Kraus, Executive Vice President and Chief Medical Officer at Otsuka Pharmaceutical Development & Commercialization, Inc., expressed optimism about the potential impact of sibeprenlimab for patients with IgAN. He highlighted the company's commitment to innovation in nephrology, aiming to address unmet needs for those with challenging conditions like IgAN. If approved, sibeprenlimab could provide important clinical benefits and ease of administration, potentially transforming the treatment landscape for this disease.

Sibeprenlimab has already received a Breakthrough Therapy designation due to its promising results in earlier trials. This designation indicates its potential to offer substantial improvements over existing therapies for IgAN. The drug's mechanism targets the 4-hit process of IgAN pathogenesis. By inhibiting APRIL, sibeprenlimab aims to disrupt the formation of immune complexes that deposit in the kidneys, potentially slowing disease progression and reducing the need for kidney transplants.

IgAN primarily affects young adults between the ages of 20 and 40 and is characterized by the build-up of Gd-IgA1 complexes in the kidneys. These immune complexes contribute to kidney damage and can lead to ESKD. Research into IgAN continues to highlight the importance of developing therapies that address the underlying causes of the disease, as current treatments often do not fully prevent progression to ESKD.

APRIL, a cytokine in the tumor necrosis factor family, is central to the disease's progression by promoting the survival and transformation of B cells, which produce the harmful Gd-IgA1. Sibeprenlimab's ability to neutralize APRIL presents a promising therapeutic strategy.

Otsuka Pharmaceutical Co., Ltd. is dedicated to advancing healthcare through innovative solutions in both pharmaceuticals and nutraceuticals. Their mission focuses on improving overall well-being with products and services backed by scientific research. Sibeprenlimab's development, if successful, could mark a significant step forward in the treatment of IgAN, offering new hope for patients facing this challenging disease.

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